EP3227447A4 - Arn guide comportant des modifications chimiques - Google Patents

Arn guide comportant des modifications chimiques Download PDF

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Publication number
EP3227447A4
EP3227447A4 EP15866342.7A EP15866342A EP3227447A4 EP 3227447 A4 EP3227447 A4 EP 3227447A4 EP 15866342 A EP15866342 A EP 15866342A EP 3227447 A4 EP3227447 A4 EP 3227447A4
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EP
European Patent Office
Prior art keywords
guide rna
chemical modifications
chemical
modifications
rna
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Granted
Application number
EP15866342.7A
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German (de)
English (en)
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EP3227447A1 (fr
EP3227447B1 (fr
Inventor
Daniel E. RYAN
Douglas J. Dellinger
Jeffrey R. Sampson
Robert Kaiser
Joel Myerson
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Agilent Technologies Inc
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Agilent Technologies Inc
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First worldwide family litigation filed litigation Critical https://patents.darts-ip.com/?family=56092183&utm_source=google_patent&utm_medium=platform_link&utm_campaign=public_patent_search&patent=EP3227447(A4) "Global patent litigation dataset” by Darts-ip is licensed under a Creative Commons Attribution 4.0 International License.
Application filed by Agilent Technologies Inc filed Critical Agilent Technologies Inc
Priority to EP24165113.2A priority Critical patent/EP4400584A3/fr
Publication of EP3227447A1 publication Critical patent/EP3227447A1/fr
Publication of EP3227447A4 publication Critical patent/EP3227447A4/fr
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    • C—CHEMISTRY; METALLURGY
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    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/111—General methods applicable to biologically active non-coding nucleic acids
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H21/00—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
    • C07H21/02—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with ribosyl as saccharide radical
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    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
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    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/90—Stable introduction of foreign DNA into chromosome
    • C12N15/902—Stable introduction of foreign DNA into chromosome using homologous recombination
    • C12N15/907—Stable introduction of foreign DNA into chromosome using homologous recombination in mammalian cells
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    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22—Ribonucleases [RNase]; Deoxyribonucleases [DNase]
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12Q1/00—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions
    • C12Q1/68—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions involving nucleic acids
    • C12Q1/6876—Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]
    • C—CHEMISTRY; METALLURGY
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/312—Phosphonates
    • C—CHEMISTRY; METALLURGY
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/321—2'-O-R Modification
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/322—2'-R Modification
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/323—Chemical structure of the sugar modified ring structure
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/323—Chemical structure of the sugar modified ring structure
    • C12N2310/3231—Chemical structure of the sugar modified ring structure having an additional ring, e.g. LNA, ENA
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/33—Chemical structure of the base
    • C12N2310/333—Modified A
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/33—Chemical structure of the base
    • C12N2310/335—Modified T or U
    • C—CHEMISTRY; METALLURGY
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/346—Spatial arrangement of the modifications having a combination of backbone and sugar modifications
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • C12N2310/3517—Marker; Tag
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/50—Physical structure
    • C12N2310/53—Physical structure partially self-complementary or closed
    • C12N2310/531—Stem-loop; Hairpin
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    • C12N2320/00—Applications; Uses
    • C12N2320/50—Methods for regulating/modulating their activity
    • C12N2320/51—Methods for regulating/modulating their activity modulating the chemical stability, e.g. nuclease-resistance
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    • C12N2330/00—Production
    • C12N2330/30—Production chemically synthesised
    • C12N2330/31—Libraries, arrays

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  • Life Sciences & Earth Sciences (AREA)
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  • Plant Pathology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Analytical Chemistry (AREA)
  • Medicinal Chemistry (AREA)
  • Cell Biology (AREA)
  • Mycology (AREA)
  • Immunology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
EP15866342.7A 2014-12-03 2015-12-03 Arn guide comportant des modifications chimiques Active EP3227447B1 (fr)

Priority Applications (1)

Application Number Priority Date Filing Date Title
EP24165113.2A EP4400584A3 (fr) 2014-12-03 2015-12-03 Arn guide avec modifications chimiques

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US201462087211P 2014-12-03 2014-12-03
US201562146189P 2015-04-10 2015-04-10
US201562256095P 2015-11-16 2015-11-16
PCT/US2015/000143 WO2016089433A1 (fr) 2014-12-03 2015-12-03 Arn guide comportant des modifications chimiques

Related Child Applications (2)

Application Number Title Priority Date Filing Date
EP24165113.2A Division EP4400584A3 (fr) 2014-12-03 2015-12-03 Arn guide avec modifications chimiques
EP24165113.2A Division-Into EP4400584A3 (fr) 2014-12-03 2015-12-03 Arn guide avec modifications chimiques

Publications (3)

Publication Number Publication Date
EP3227447A1 EP3227447A1 (fr) 2017-10-11
EP3227447A4 true EP3227447A4 (fr) 2018-07-11
EP3227447B1 EP3227447B1 (fr) 2024-04-24

Family

ID=56092183

Family Applications (2)

Application Number Title Priority Date Filing Date
EP15866342.7A Active EP3227447B1 (fr) 2014-12-03 2015-12-03 Arn guide comportant des modifications chimiques
EP24165113.2A Pending EP4400584A3 (fr) 2014-12-03 2015-12-03 Arn guide avec modifications chimiques

Family Applications After (1)

Application Number Title Priority Date Filing Date
EP24165113.2A Pending EP4400584A3 (fr) 2014-12-03 2015-12-03 Arn guide avec modifications chimiques

Country Status (8)

Country Link
US (5) US10900034B2 (fr)
EP (2) EP3227447B1 (fr)
JP (2) JP7068821B2 (fr)
KR (2) KR102629128B1 (fr)
CN (1) CN107250148B (fr)
AU (1) AU2015355546B2 (fr)
CA (1) CA2969619A1 (fr)
WO (1) WO2016089433A1 (fr)

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* Cited by examiner, † Cited by third party
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US9526784B2 (en) 2013-09-06 2016-12-27 President And Fellows Of Harvard College Delivery system for functional nucleases
US9228207B2 (en) 2013-09-06 2016-01-05 President And Fellows Of Harvard College Switchable gRNAs comprising aptamers
US9322037B2 (en) 2013-09-06 2016-04-26 President And Fellows Of Harvard College Cas9-FokI fusion proteins and uses thereof
US20150165054A1 (en) 2013-12-12 2015-06-18 President And Fellows Of Harvard College Methods for correcting caspase-9 point mutations
EP3690044B1 (fr) 2014-02-11 2024-01-10 The Regents of the University of Colorado, a body corporate Ingénierie génomique multiplexe validée ayant recours au système crispr
US10077453B2 (en) 2014-07-30 2018-09-18 President And Fellows Of Harvard College CAS9 proteins including ligand-dependent inteins
CA2969619A1 (fr) 2014-12-03 2016-06-09 Agilent Technologies, Inc. Arn guide comportant des modifications chimiques
EP3234134B1 (fr) 2014-12-17 2020-05-27 ProQR Therapeutics II B.V. Édition ciblée d'arn
CA2970683A1 (fr) 2014-12-18 2016-06-23 Integrated Dna Technologies, Inc. Compositions a base de crispr et leurs procedes d'utilisation
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US12043843B2 (en) 2015-11-04 2024-07-23 Vertex Pharmaceuticals Incorporated Materials and methods for treatment of hemoglobinopathies
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